Safety and efficacy of BEZ235 in patients with pancreatic neuroendocrine tumors (pNET)

• Register: EUCTR
• Last refreshed on: 10 July 2015
• Main ID: EUCTR2012-000675-16-NL
• Date of registration: 10/08/2012
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharma Services AG
• Public title: Safety and efficacy of BEZ235 in patients with pancreatic neuroendocrine tumors (pNET)
• Scientific title: A multicenter, two stage, phase II study, evaluating the efficacy of oral BEZ235 plus best supportive care (BSC) versus placebo plus BSC in the treatment of patients with advanced pancreatic neuroendocrine tumors (pNET) after failure of mTOR inhibitor therapy
• Date of first enrolment: 13/09/2012
• Target sample size: 150
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2012-000675-16
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 2

Countries of recruitment

Austria, Belgium, Germany, Italy, Korea, Republic of, Netherlands, Spain, United Kingdom, United States

Contacts

• Name: Clinical Trial Information Desk
• Affiliation: Novartis Pharma Services AG

• Name: Clinical Trial Information Desk
• Affiliation: Novartis Pharma Services AG

Key inclusion & exclusion criteria

Inclusion criteria:
Inclusion Criteria: Unresectable or metastatic, histologically confirmed low or intermediate grade pancreatic neuroendocrine
tumor with radiological evidence of disease progression since last treatment. Refractory disease to treatment with mTOR
inhibitor, Measurable disease per RECIST Version 1.1 using Computed Tomography (CT) or Magnetic Resonance Imaging
(MRI), Prior or concurrent therapy with SSA is permitted;a stable dose at least 2 months prior to study start and must
continue on the stable dose while receiving study treatment; Other protocol defined inclusion/exclusion criteria may apply.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 100
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 50

Exclusion criteria:
Exclusion Criteria: Previous treatment with any PI3K or AKT inhibitor, Discontinuation prior mTOR inhibitor therapy due to
toxicity, Poorly differentiated neuroendocrine carcinoma, highgrade neuroendocrine carcinoma, adenocarcinoid, goblet cell
carcinoid and small cell carcinoma, Cytotoxic chemotherapy, targeted therapy, immunotherapy, radiotherapy, or major
surgery within 4 weeks prior to enrolment in the study, Hepatic artery embolization within the last 6 months (1 month if there are other sites of measurable disease), or cryoablation/ radiofrequency ablation of hepatic metastasis within 2 months of enrollment More than 3 prior systemic treatment regimens, Other protocol defined inclusion/exclusion criteria may apply


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Cancer [C04]

Adult patient in advanced pancreatic neuroendocrine tumors
MedDRA version: 14.1 Level: LLT Classification code 10068916 Term: Pancreatic neuroendocrine tumor metastatic System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

Intervention(s)

Product Name: BEZ235
Product Code: BEZ235
Pharmaceutical Form: Powder for oral suspension
INN or Proposed INN: BEZ235
CAS Number: 1028385-32-1
Current Sponsor code: BEZ235
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 200-
Pharmaceutical form of the placebo: Powder for oral suspension
Route of administration of the placebo: Oral use

Product Name: BEZ235
Product Code: BEZ235
Pharmaceutical Form: Powder for oral suspension
INN or Proposed INN: BEZ235
CAS Number: 1028385-32-1
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 300-
Pharmaceutical form of the placebo: Powder for oral suspension
Route of administration of the placebo: Oral use

Product Name: BEZ235
Product Code: BEZ235
Pharmaceutical Form: Powder for oral suspension
INN or Proposed INN: BEZ235
CAS Number: 1028385-32-1
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 400-
Pharmaceutical form of the placebo: Powder for oral suspension
Route of administration of the placebo: Oral use

Primary Outcome(s)

Secondary Objective: To assess the safety and tolerability of BEZ235 therapy
To evaluate the efficacy of BEZ235 per modified RECIST v1.1 (Overall Response Rate, Disease Control Rate, Duration of Response )

Timepoint(s) of evaluation of this end point: up to approx 18 months

Primary end point(s): PFS rate at 16 weeks according to local radiological assessment per modified RECIST v1.1

Main Objective: To estimate the efficacy of BEZ235 in adult patients with advanced (unresectable or metastatic) pNET (PFS)

Secondary Outcome(s)

Secondary end point(s): Frequency and severity of adverse events;other safety data as considered appropriate
Overall Response Rate, Disease Control Rate, Duration of Response )

Timepoint(s) of evaluation of this end point: up to approx. 18 months for all secondary endpoint

Secondary ID(s)

2012-000675-16-AT

CBEZ235F2201

Source(s) of Monetary Support

Novartis Pharma Services AG